Literature DB >> 33485465

Considerations for Systemic Use of Gene Therapy.

Barry J Byrne1, Manuela Corti2, Francesco Muntoni3.   

Abstract

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Year:  2021        PMID： 33485465      PMCID： PMC7854351          DOI： 10.1016/j.ymthe.2021.01.016

Source DB:  PubMed          Journal:  Mol Ther        ISSN： 1525-0016            Impact factor:   11.454


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  6 in total

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Journal:  J Pediatr       Date:  2020-11-28       Impact factor: 4.406

Review 2.  Satellite cells and the muscle stem cell niche.

Authors:  Hang Yin; Feodor Price; Michael A Rudnicki
Journal:  Physiol Rev       Date:  2013-01       Impact factor: 37.312

Review 3.  Onasemnogene Abeparvovec: First Global Approval.

Authors:  Sheridan M Hoy
Journal:  Drugs       Date:  2019-07       Impact factor: 9.546

4.  Evidence for cardiomyocyte renewal in humans.

Authors:  Olaf Bergmann; Ratan D Bhardwaj; Samuel Bernard; Sofia Zdunek; Fanie Barnabé-Heider; Stuart Walsh; Joel Zupicich; Kanar Alkass; Bruce A Buchholz; Henrik Druid; Stefan Jovinge; Jonas Frisén
Journal:  Science       Date:  2009-04-03       Impact factor: 47.728

5.  SOD1 Suppression with Adeno-Associated Virus and MicroRNA in Familial ALS.

Authors:  Christian Mueller; James D Berry; Diane M McKenna-Yasek; Gwladys Gernoux; Margaret A Owegi; Lindsay M Pothier; Catherine L Douthwright; Dario Gelevski; Sarah D Luppino; Meghan Blackwood; Nicholas S Wightman; Derek H Oakley; Matthew P Frosch; Terrence R Flotte; Merit E Cudkowicz; Robert H Brown
Journal:  N Engl J Med       Date:  2020-07-09       Impact factor: 91.245

6.  European ad-hoc consensus statement on gene replacement therapy for spinal muscular atrophy.

Authors:  Janbernd Kirschner; Nina Butoianu; Nathalie Goemans; Jana Haberlova; Anna Kostera-Pruszczyk; Eugenio Mercuri; W Ludo van der Pol; Susana Quijano-Roy; Thomas Sejersen; Eduardo F Tizzano; Andreas Ziegler; Laurent Servais; Francesco Muntoni
Journal:  Eur J Paediatr Neurol       Date:  2020-07-09       Impact factor: 3.140
  6 in total
  2 in total

1.  AAV vector manufacturing process design and scalability - Bending the trajectory to address vector-associated immunotoxicities.

Authors:  J Fraser Wright
Journal:  Mol Ther       Date:  2022-05-20       Impact factor: 12.910

2.  Effect of CpG Depletion of Vector Genome on CD8+ T Cell Responses in AAV Gene Therapy.

Authors:  Thais B Bertolini; Jamie L Shirley; Irene Zolotukhin; Xin Li; Tsuneyasu Kaisho; Weidong Xiao; Sandeep R P Kumar; Roland W Herzog
Journal:  Front Immunol       Date:  2021-05-31       Impact factor: 7.561
  2 in total

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